BIO 346 Human Biology (K. Kalthoff)

References to Topic 11: Human Gene Therapy

Easy Readings

Anderson W.F. (1995) Gene therapy. Scientific American Sep. 1995: 124-128.
Bank A. (1996) Human somatic gene therapy. BioEssays 18: 999-1007
Friedmann T. (1997) Overcoming the obstacles to gene therapy. Scientific American June 1997: 96-101
Marshall E. (1995) Gene therapy's growing pains. Science 269: 1050-1055
Marshall E. (1999) Gene therapy death prompts review of adenovirus vector. Science 286: 2244-2245
Tsien J.Z. (2000) Building a brainier mouse. Scient. Amer. April 2000: 62-68

Intermediate Readings

Friedmann T. (1992) A brief history of gene therapy. Nature Genetics 2: 93-98.
Jones III J.P., Kierlin M.N., Coon R.G., Perutka J., Lambowitz A.M. and Sullenger B.A. (2005) Retargeting mobile group II introns to repair mutant genes. Molecular Therapy 11: 687-694
Merkle CJ, Montgomery DW. (2003) Gene therapy with vascular endothelial growth factor reduces angina. J Cardiovasc Nurs. 18: 38-43.
Rosenberg S.A. (2001) Progress in human tumor immunology and immunotherapy. Nature 411: 380-384
Sawyers C. (2004) Targeted cancer therapy. Nature 432: 294-297
Tsien J.Z. (2000) Linking Hebb's coincidence-detection to memory formation. Curr. Opin. Neurobiol. 10: 266-273
Yamanaka S. and Blau H.M. (2010) Nuclear reprogramming to a pluripotent state by three approaches. Nature 465: 704-712

Advanced Readings

Blaese R.M. et al. (1995) T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 270: 475-480
Boland M. et al. (2009) Adult mice generated from induced pluripotent stem cells. Nature 461: 91-94
Bordignon C. et al. (1995) Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- Immunodeficient patients. Science 270: 470-475
Campbell K.P. (1995) Three Muscular Dystrophies: loss of cytoskeleton-extracellular matrix linkage. Cell 80: 675-679.
Cartier N. et al. (2009) Hematopoietic stem cell gene therapy with lentiviral vector in X-linked adrenoleukodystrophy. Science 326: 818-823
Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A. (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 288: 669-672.
Cox G.A., Cole N.M., Matsumura K., Phelps S.F., Hauschka S.D., Campbell K.P., Faulkner J.A. and Chamberlain J.S. (1993) Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity. Nature 364: 725-729.
Hacein-Bey-Abina S. et al. (2003) LMO2-associated clonal T cell prolifereation in two patients after gene therapy for SCID-X1. Science 302: 415-419
Gore A. et al. (2011) Somatic coding mutations in human induced pluripotent stem cells. Nature 471: 63-

Hussein S.M. et al. (2011) Copy number variation and selection during reprogramming to pluripotency. Nature 471: 58-62
Jaglo-Ottosen K.R., Gilmour S.J., Zarka D.G., Schabenberger O., Thomashow M.F. (1998) Arabidopsis CBF1 overexpression induces COR genes and enhances freezing tolerance. Science 280: 104-106.
Jiao S., Gurevich V. and Wolff J.A. (1993) Long-term correction of rat model of Parkinson's disease by gene therapy. Nature 362: 450-453.
Kaji K. et al. (2009) Virus-free induction of pluripotency and subsequent excision of reprogramming factors. Nature 458: 771-775
Kay M.A., Rothenberg S., Landen C.N., Bellinger D.A., Leland F., Toman C., Finegold M., Thompson A.R., Read M.S., Brinkhous K.M. and Woo S.L.C. (1993) In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs. Science 262: 117-119.
Laurent L.C. et al. (2011) Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESC and iPSCs during reprogramming and time in culture. Cell Stem Cell 8: 106-118
Lister R. et al. (2011) Hotspots of aberrant epigenomic reprogramming in human induced pluripotent stem cells. Nature doi:10.1038/nature 09798
Losordo D.W., Vale P.R., Symes J.F., Dunnington C.H., Esakof D.D., Maysky M., Ashare A.B.,Lathi K. and Isner J.M. (1998) Gene therapy for myocardial angiogenesis: initial clinical results with direct myocardial injection of phVEGF165 as sole therapy for myocardial ischemia. Circulation 98(25): 2800-2804
Manos W.L. et al (2010) Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 5: 618-630
Obokata H. et al. (2014) Stimulus-triggered fate conversion of somatic cells into pluripotency. Nature 505: 641-647
Okita K., Ichisaka T. and Yamanaka S. (2007) Generation of germline-competent induced pluripotent stem cells. Nature 448: 313-318
Park I.-H. et al. (2008) Reprogramming of human somatic cells to pluripotency with defined factors. Nature 451: 141-146
Raya A. et al. (2009) Disease-corrected hematopoietic progenitors from Franconi anaemia induced pluripotent stem cells. Nature 460: 53
Rais Y. et al. (2013) Deterministic direct reprogramming of somatic cells to pluripotency. Nature 502: 65-70.
Rideout III W.M., Hochedlinger K., Kyba M., Daley G.Q. and Jaenisch R. (2002) Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy. Cell 109: 17-27
Takahashi K. and Yamanaka S. (2006) Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors.   Cell 126: 663-676
Vale P.R., Losordo D.W., Milliken C.E., Maysky M., Esakof D.D., Symes J.F. and Isner J.M. (2000) Left ventricular electromechanical mapping to assess efficacy of phVEGF(165) gene transfer for therapeutic angiogenesis in chronic myocardial ischemia. Circulation 102(9): 965-974
Woltjen K. et al. (2009) piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells. Nature 458: 766
Yu J. et al. (2007) Induced pluripotent stem cell lines derived from human somatic cells. Science 318: 1917-1920
Zhao X. (2009) iPS cells produce viable mice through tetraploid complementation. Nature 461: 86-90
Zhou Q., Brown J., Kanarek A., Rajagopa J. and Melton D.A. (2008) In vivo reprogramming of adult pancreatic exocrine cells to beta cells. Nature 455: 627
Zwaka TP and Thomson JA. (2003) Homologous recombination in human embryonic stem cells. Nat Biotechnol. Feb 10; [epub ahead of print]

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